New Therapy for a Rare Syndrome

September 8th, 2010

New Therapy for a Rare Syndrome

Larry Husten, PHD

A team of French and Belgian investigators have found a promising new treatment for a rare inherited disorder that often leads to vascular dissection or rupture. In a report published online in the Lancet, Kim-Thanh Ong and colleagues compared the effect of celiprolol, a beta-blocker with beta-2 agonist properties, with placebo in 53 patients with Ehlers-Danlos syndrome. After 47 months of follow-up, the trial was stopped early when the investigators observed a significant reduction in ruptures or dissections in the celiprolol group compared with the placebo group (5 vs. 14).

In an accompanying editorial, Benjamin Brooke writes that the study “represents a substantial breakthrough in the evidence-based management of the syndrome. Celiprolol is an inexpensive well-tolerated drug available to treat patients with vascular Ehlers-Danlos syndrome worldwide.”

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September 8th, 2010